Background Multiple myeloma (MM) is a heterogeneous disease that is most frequently diagnosed in the elderly. Therefore, data on clinical characteristics and outcomes in the young population are scarce and it is recognized that it remains incurable even in this group of patients. We present here the outcomes of patients under 40 years old cohort in Latin-American countries. On behalf of GELAMM (Grupo de Estudio Latino-Americano de Mieloma Múltiple). Methods Retrospective international multicenter cohort study. We analyzed MM patients under 40 years old who received treatment in 6 Latin-American countries, between 2010 and 2018. Demographics and disease features were analyzed using descriptive statics. We examined treatment characteristics and response rates. The overall survival (OS) of the entire cohort was analyzed using Kaplan-Meier curves. Results Eighty-six patients of 6 countries were analyzed (Table1). The mean age was 35.4 years old, and 60% were male. The most frequent monoclonal component type was IgG followed by light chain MM. Risk determined by ISS was distributed in almost equal percentages. The most frequent cytogenetic alteration was the t (4;14) that was found in four patients out of 25 evaluated. The missing data were greater than 70%. Skeleton-related events were the most frequent clinical feature, followed by anemia and renal failure. Plasmacytomas and fractures were present in more than 20 percent of cases. With regard to treatment, VCD / CyBorD was the most used regimen, followed by VTD. The overall response rate (ORR) was 63%. Fifty-three patients received high dose therapy and autologous stem cell transplantation (62%). Only 8% received post-transplant consolidation, and 45% received maintenance therapy. The median OS of the entire cohort was 45 months, and a plateau in the survival curve was not observed, suggesting that patients continue relapsing over the time. Conclusion In this Latin American multicenter study, we found that the young population with MM has similar presentation characteristics to those of elderly patients. A significant amount of information is lost regarding the risk characterization, especially in regard with cytogenetics. With respect to treatment, less than half of the patients achieve very good partial response or better. It is striking that more than a third of this young patients did not access to high doses of chemotherapy and bone marrow transplantation. Maintenance therapy is offered to less than half patients. The median OS is lower than in other series of patients younger than 40 years, even than in the elderly cohorts. Prospective multicentric studies are required to elucidate the behavior of the disease in this group of patients. Disclosures Peña: Pfizer: Membership on an entity's Board of Directors or advisory committees; Janssen: Other: Congress inscription and flights; Biotoscana: Other: Congress inscription and flights; Novartis: Other: Congress inscription and flights; Tecnofarma: Other: Congress inscription and flights; Roche: Other: Congress inscription and flights. Rojas:Novartis: Membership on an entity's Board of Directors or advisory committees; Pfeizer: Membership on an entity's Board of Directors or advisory committees; Abbvie: Membership on an entity's Board of Directors or advisory committees; Roche: Membership on an entity's Board of Directors or advisory committees. Abello:Takeda: Other: Participation in advisory board meeting. Gomez-Almaguer:Takeda: Consultancy, Speakers Bureau; Celgene: Consultancy, Speakers Bureau; Amgen: Consultancy, Speakers Bureau; Janssen: Consultancy, Speakers Bureau; Teva: Consultancy, Speakers Bureau.
Introduction Compliance with Multiple Myeloma (MM) recommendations regarding diagnosis and treatment is highly variable worldwide, outside clinical trials. This survey among hematologists from Latin America (LA) aims to describe real access to diagnostic and prognostic analyses and first line treatment for newly diagnosed MM (NDMM). Objectives To describe the access to diagnostic and prognostic tests and first line treatment options for MM in LA. To compare public versus private access to tests and therapies. Methods This is a multicenter cross-sectional study. A 16-question survey focusing on demographic characteristics of physicians, centers, and standard of care practices for NDMM was emailed to 182 hematologists from 11 LA countries. (Fig 1) The survey was open from Dec/17-Feb/18. Results We received 103 completed questionnaires (56.6%) from 8 countries: Argentina (45), Uruguay (28), Chile (15), Paraguay (6), Peru (3), Costa Rica (2), Mexico (2), Venezuela (2). Most physicians (85/103) work in private and public institutions; the majority (64.7%) treat benign and malignant diseases, 30% mainly malignant diseases, and 4.9% mainly plasma cell disorders. Access to diagnostic tests is shown in Table 1. In > 20% of public hospitals there is no access to serum protein electrophoresis (SPEP), serum immunofixation (IFX) or serum immunoglobulins quantification (Igs); in 57.3% serum Free Light Chain (sFLC) assay is not done. Lack of access to Fluorescent in situ hybridization (FISH) is 67%, Computed Tomography (CT) scan 23.5%, Magnetic Resonance Imaging (MRI) 45% and Positron Emission Tomography (PET/CT) 66.5%. In private centers, lack of access to SPEP is <5%, IFX 10%, Igs 15%, sFLC 16.5%, FISH 33%, CT scan 5%; MRI 7.3% and PET/CT 20%. Thalidomide, Bortezomib and autologous stem cell transplant (ASCT) are available in all reporting countries, in public and private institutions. Lenalidomide is reported commercially available by 97.9% of physicians, Melphalan 92.7%, Daratumumab 68%, Pomalidomide 67%, Carfilzomib 60%, Ixazomib 18%. (Table 2) Nevertheless, due to reimbursement policies not all patients have access to these drugs, as reflected in treatment choices. In private institutions, 86.5% report treating ASCT-eligible patients upfront with Bortezomib-based triplets versus 51.6% in public hospitals [OR 5.72 (IC95% 2.32-14.7) p <0.001). Cyclophosphamide-Thalidomide-Dexamethasone (CTD) is used in the public setting by 40.3% (Table 3). Firstline choice for high risk MM (HRMM) is Bortezomib combinations in all private centers but in only 74.4% of public institutions (p<0.001). ASCT ineligible patients: the most used upfront regimen is Cyclophosphamide-Bortezomib-Dexamethasone (CyBorD) in private centers and CTD in public hospitals (Table 4). For HRMM, Bortezomib-based triplets is the first choice in 88.57% and 65.51% in private and public settings, respectively [OR 4.09 (CI95% 1.57-11.16) p=0.0011). Most physicians indicate maintenance treatment, mainly until progression/intolerance.(Table 5) Lenalidomide or Bortezomib are used in private centers in all countries, but for Venezuela. Only Thalidomide and Dexamethasone are available in public hospitals from 6/8 countries. Lenalidomide or Bortezomib maintenance approval require special authorization in most countries, delaying its initiation. Discussion This study shows real word data regarding the challenges LA faces in the care of MM patients. Access to recommended diagnostic and prognostic tests is deficient, particularly in the public setting. Diagnosis, risk assessment and response evaluation are, therefore, inaccurate. In public hospitals, >50% of patients have no access to adequate imaging evaluation, being screened by X-rays. Regardless of commercial availability, real access to novel drugs is limited, particularly in the public setting. This causes an ethical dilemma for physicians, which must treat patients depending on the health care provider and reimbursement policies rather than based on evidence. One limitation of this work is the high percentage of unanswered surveys. Conclusion LA is far from complying with international recommendations for MM approach. The most striking finding is the great difference between public and private centers in all variables. This gap is likely to translate into differences in survival, which is greatly concerning. Solving these inequities should be a priority. Disclosures No relevant conflicts of interest to declare.
Background: Philadelphia-negative Myeloproliferative Neoplasms (Ph-MPN) are chronic hematological disorders characterized by the overproduction of one or more mature myeloid blood cell lineages. Classical Ph-MPN are Polycythemia Vera (PV), Essential Thrombocytopenia (ET) and Myelofibrosis (MF). The diagnosis includes clinical, histological and molecular features. There are not data from Chile. The aim of this study is to determinate epidemiological, clinical, diagnostic and therapeutic characteristics of Ph-MPN in our country. Methods: Descriptive and retrospective study. We reviewed the database of the Molecular Biology Laboratory at the Hospital del Salvador, a national reference laboratory, from 2012 to 2017. All patients referred as Ph-MPN were included. We reviewed the clinical records to obtain clinical information. Results: Clinical data was obtained from 468 cases from 12 public hospitals in Chile. Median age at diagnosis was 70 years. Female to Male ratio= 1,15:1, without significant differences between Ph-MPNs. ET was the most frequently Ph-MNP found, accounting for 49,4% of all Ph-MPN, followed by PV (37%) and MF (10,4%). A 66,2% of ET was JAK2 V617F+. Bone marrow biopsy was performed in 35% of ET cases. Only 7,8% had cytogenetic study. Splenomegaly was found in 8%. Thrombosis was observed in 23,8%. The median platelet count was 842x109/L. All patients received hydrea +/- aspirin or oral anticoagulation. Of the total of PV, 86,6% was JAK2+. Bone marrow biopsy was performed in a quarter of the cases. Thrombosis frequency was 14,5%. A 29% had splenomegaly. Median hemoglobin level was 18 gr/dl. All patients were treated with aspirin +/- phlebotomy and about half of them required cytoreduction. Two patients were refractory to hydrea and used ruxolitinib as second line treatment. A 63,3% of the MF were JAK-2+. Bone marrow biopsy was performed in 59% and 20% had a cytogenetic study. Only one fifth of patients had LDH measurement at diagnosis. Splenomegaly was observed in 75,5% of cases. Thrombosis frequency was 13%. Anemia was the most frequent finding in complete blood count. The treatments were heterogeneous, including hydrea, EPO, thalidomide/prednisone, danazol and ruxolitinib. Discussion: TE was the most common Ph-MPN. The epidemiological and blood count findings were similar to the data reported in the literature. It is important to note that with the 2016 WHO classification new criteria, some of patients diagnosed with ET, now will be in PV cathegory (21 patients in our serie). The distribution of JAK2V617F+ in Ph-MPN was similar to the published data, except for PV, in which we found a lower percentage of JAK2+. Thrombosis were lower than the data reported for PV. It is worrisome that bone marrow biopsy and cytogenetic study were performed only in a low percentage of the patients. The treatment strategies were heterogeneous and not standardized among the participating centers. These findings reveal a lack in the use of the diagnostic tools for Ph-MPN. It is important to improve clinical and molecular characterization of these patients in order to guide available therapeutic alternatives in our country. Disclosures No relevant conflicts of interest to declare.
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